DESCRIPTION (taken from the application) Gene therapy approaches have made important strides in the last several years. This is in large part due to the technical advances in vector delivery approaches. A number of preclinical studies has demonstrated reduced toxicity, with prolonged and therapeutic levels of clinically relevant gene products from various tissues. This is likely to lead to early success in new clinical trials under development. However, not all problems have been solved and there are many diseases with complex pathophysiology for which gene therapy has yet to show promise. Moreover, there are new safety obstacles that have arisen that will likely preclude treating less severe medical conditions. The meeting will highlight recent successes and barriers that still challenge the gene therapy community as it relates to basic science, preclinical animal studies, and clinical trials. The meeting will be held approximately 6 months away from the American Society for Gene Therapy (ASGT) meeting and will feature important new results that have occurred during the intervals of the yearly ASGT meetings. The meeting will also offer a format for research summaries as well as new primary data for newcomers to the field. The environment will be conducive for young investigators to interact directly with more senior investigators. This interaction is likely to allow trainees to find new mentors for advanced training (e.g., senior students who wish to pursue post-doctoral training; therefore, student participation is a critical component of the meeting.The plenary speakers blend a mix of senior, middle career, and young rising stars. The senior scientists who we anticipate will have new important accomplishments in 2001 were selected to help attract trainees to the conference. Session chairs were selected from the senior leaders in the specific target areas. All of the speakers (including the younger investigators) were selected on their recent past record of superb scientific accomplishment and verbal articulation. The younger scientists were included to add more depth and present fresh new ideas. We have selected 7 more "junior" speakers who have presented outstanding data at a session during the last ASGT meeting.